Ohlfest and Moertel are now examining a new “vaccine” for three types of recurrent brain tumors: glioblastoma, medulloblastoma, and ependymoma. While many vaccines work to prevent disease, this one treats existing disease.

Customized by using a patient’s own immune cells, the therapy is designed to stimulate an immune response that destroys tumor stem cells—the parent cells responsible for tumor growth.

The Phase I clinical trial began at University of Minnesota Medical Center, Fairview and University of Minnesota Amplatz Children’s Hospital in late 2010. It includes both children and adults who have recurrent brain tumors despite extensive treatment with surgery, chemotherapy, and radiation.

The team recently enrolled the study’s first participant. “This person couldn’t have waited another year,” Ohlfest says.

Early investment = Quicker results

Working with Ohlfest and Moertel on the clinical trial is Walter Low, Ph.D., professor in the Department of Neurosurgery, and a 20-member lab staff. The research grew out of a strategic approach developed by John Wagner, M.D., the Hageboeck/Children’s Cancer Research Fund Chair in Pediatric Cancer Research. Five years ago, Wagner put together a core group—including Ohlfest, Moertel, and David Largaespada, Ph.D.—to lead the fight against malignant brain tumors. He made sure they got the funding they needed to transform the way brain tumors were treated.

“I knew that breakthrough research would only come about if we invested in the research,” Wagner says.

The crucial early investments came from Children’s Cancer Research Fund, a 30-year partner in supporting the University’s pioneering research to prevent, treat, and cure childhood cancer. Brain tumor research is a priority for the organization, says CEO John Hallberg.

“Our generous donors provided fast access to unrestricted funds so University scientists could accelerate their work,” he says. “This has yielded positive clinical results for patients and additional dollars in funding.”

A special interest

Longtime Children’s Cancer Research Fund donors John and Jean Hedberg have taken a special interest in this research. Their granddaughter, Anda Moettus, had a brain tumor at age 4. Extensive treatment saved her life but left her with significant disabilities. Now 19, Anda has graduated from high school and is learning to live independently.

“Our family became interested in the University’s research to advance the treatment of brain cancer and to reduce the problems for people who survive,” John Hedberg explains.

Hallberg credits the Hedberg family foundation with driving the brain tumor program.

For Ohlfest, creating effective therapies for people with brain tumors can’t happen soon enough. In graduate school he dedicated himself to fighting brain cancer, realizing that a lack of drugs—and research—was costing people’s lives. One of those was his grandmother, who died from ovarian cancer that metastasized to her brain.

“Brain tumors come back with extreme fury,” Ohlfest says. “Our work is never enough—not until this is cured.”